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Intrabone Infusion of Umbilical Cord Blood Stem Cells

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01711788
First Posted: October 22, 2012
Last Update Posted: October 5, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
Michel Duval, MD
Pierre Teira, MD
Sonia Cellot, MD, PhD
Isabelle Louis, PhD
Elie Haddad, MD, PhD
Marie-France Vachon, MScN
Marion Cortier, PhD
Information provided by (Responsible Party):
Henrique Bittencourt, MD, PhD, St. Justine's Hospital
  Purpose
The purpose of this study is to determine if the method of intrabone infusion of hematologic stem cells can increase and accelerate hematopoietic reconstitution after umbilical cord blood transplantation in pediatric patients.

Condition Intervention Phase
Hematopoietic Stem Cell Transplantation Procedure: Intrabone infusion of umbilical cord blood stem cells Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A New Approach to Improve Long-term Hematopoietic Recovery After Allogeneic Umbilical Cord Blood Transplantation in Children - Intrabone Infusion of Umbilical Cord Blood Stem Cells

Further study details as provided by Henrique Bittencourt, MD, PhD, St. Justine's Hospital:

Primary Outcome Measures:
  • Platelet recovery rate [ Time Frame: at 100 days post- transplantation ]
    First of seven days of untransfused platelet count higher than 20 x 10^9/L


Secondary Outcome Measures:
  • Neutrophil recovery rate [ Time Frame: at 60 days post- transplantation ]
    First of three days of absolute neutrophil count equal or higher than 0.5 x 10^9/L

  • Immunological reconstitution [ Time Frame: at 30, 60, 100, 180, and 360 days post- transplantation ]
    Total number of T cells (and subpopulations), B and NK (natural killer) cells in peripheral blood at different time-points

  • Donor chimerism rate [ Time Frame: at 30, 60,100, and 180 days post-transplantation ]
    Percentage of donor(s) cells in peripheral blood at different time-points

  • Acute GVHD (grade 2-4) rate [ Time Frame: at 180 days ]
    Incidence of grade II-IV acute GVHD (Graft versus Host Disease)

  • Infection rate (bacterial, viral, fungal and parasitic) [ Time Frame: at 180 days post-transplantation ]
    Clinical and microbiological documented infections will be reported according to anatomic site, date of onset and microorganism

  • Event-free and overall survival [ Time Frame: at 2 years ]
    Event-free survival is defined as the time interval between transplantation and relapse, graft rejection, death or last follow-up, whichever occurs first; Overall survival is defined as the time between transplantation and death or last follow-up

  • Adverse infections (grade and frequency) [ Time Frame: at one month post-transplantation ]
    Toxicity will be assessed using the Common Terminology Criteria for Adverse Events v4.0

  • chronic GVHD [ Time Frame: at 2 years post-transplantation ]
    Incidence of chronic GVHD (Graft versus Host Disease) will be scored according to NIH consensus on chronic GVHD


Enrollment: 15
Study Start Date: November 2012
Study Completion Date: October 3, 2017
Primary Completion Date: October 3, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intrabone umbilical cord blood tranplant
Intrabone infusion of umbilical cord blood stem cells
Procedure: Intrabone infusion of umbilical cord blood stem cells

Detailed Description:

Umbilical cord blood transplantation (UCBT) has been increasingly used to treat malignant and non-malignant haematological, immunodeficiency and some metabolic diseases. UCBT offers the advantages of easy procurement, no risk to donors, a reduced risk of transmitting infections, immediate availability of cryopreserved units, and acceptable partial HLA mismatches. However, patients treated with UCBT show delayed hematopoietic and immunological recoveries, have higher rates of infection, and relapse from the original malignant disease, which can all lead to life threatening problems. UCBT can also result in a higher rate of graft failure compared to other hematopoietic stem cell transplantation (HSCT) sources. The problem of a slower hematopoietic recovery post-UCBT has been addressed using a number of different approaches in adult patients.In adults, use of intrabone injection of cord blood results in a faster hematopoietic recovery in a phase II study. However, there is no clinical trial in pediatric patients.

This study is addressed to determine if a change in the cord blood stem cell infusion method can increase and accelerate hematopoietic reconstitution after UCBT in pediatric patients.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • One to 21 years of age;
  • More than 10 kg in weight;
  • Diagnosis of hematopoietic disorders (malignant or not) with an indication for hematopoietic stem cell transplantation;
  • Absence of an HLA-identical related donor;
  • Availability of a single cord blood (CB) with at least 3 x 10^7 nucleated cells (NCs)/kg (if HLA identical or 1 HLA-mismatch) or at least 4 x 10^7 NCs/kg (if a 2 HLA-mismatch) at freezing. Use of two CB units ("double cord transplant") will be allowed provided that: 1) a single CB unit fulfilling the above criteria is not available; 2) a maximum of 2 HLA mismatch is present for each CB unit; and 3) a minimum of 4 x 10^7 NCs/kg (as the sum for both CB units) is present at freezing.
  • A myeloablative-conditioning regimen;
  • A Lansky (for patients less than 16 years of age) or Karnofsky (for patients more than 16 years of age) score equal to or higher than 70%.
  • Adequate organ function as follows:
  • Cardiac (ejection fraction > 50%);
  • Renal (serum creatinine within the normal range for age, and creatinine clearance or a GFR > 70 ml/min/1.73m2);
  • Hepatic (AST or ALT < 5 x upper limit of normal for age);
  • Pulmonary (FEV1, FVC, and DLCO ≥ 50% by pulmonary function tests or, in children unable to cooperate, no sign of dyspnea at rest, no exercise intolerance, no supplementary oxygen therapy, and a normal pulmonary radiography or pulmonary scan);
  • No sign of uncontrolled systemic bacterial, fungal or viral infection;
  • Written informed consent by the patient or his/her legal guardian

Exclusion Criteria:

  • Non-myeloablative conditioning;
  • Pregnancy or breastfeeding;
  • HIV positive serology;
  • Bone disease (e.g. osteopetrosis, osteogenesis imperfecta)
  • Previous autologous or allogeneic hematopoietic stem cell transplantation performed up to one year before enrolment, except in the case of non-engraftment or early rejection of a previous allogeneic stem cell transplantation.
  • Active skin infection at the site of intrabone injection.
  • History of intolerance/allergy to sedation medications or local anesthetics.
  • Contraindication to sedation
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01711788


Locations
Canada, Quebec
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada, H3C1T5
Sponsors and Collaborators
Henrique Bittencourt, MD, PhD
Michel Duval, MD
Pierre Teira, MD
Sonia Cellot, MD, PhD
Isabelle Louis, PhD
Elie Haddad, MD, PhD
Marie-France Vachon, MScN
Marion Cortier, PhD
Investigators
Principal Investigator: Henrique Bittencourt, MD, PhD St. Justine's Hospital
  More Information

Responsible Party: Henrique Bittencourt, MD, PhD, Hematologist - Oncologist, St. Justine's Hospital
ClinicalTrials.gov Identifier: NCT01711788     History of Changes
Other Study ID Numbers: IB-UCBT
First Submitted: October 18, 2012
First Posted: October 22, 2012
Last Update Posted: October 5, 2017
Last Verified: October 2017

Keywords provided by Henrique Bittencourt, MD, PhD, St. Justine's Hospital:
Hematopoietic stem cells
Umbilical cord blood transplantation
Intrabone infusion
Platelet recovery
Neutrophil recovery
Pediatric patients